E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Treatment of iron deficient non anaemic women with ferric carboxymaltose. |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 12.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10022970 |
E.1.2 | Term | Iron deficiency |
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E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To assess the efficacy of a single intravenous (IV) administration of FCM (1,000 mg) compared with placebo in improving fatigue symptoms in IDNA women of child bearing age. |
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E.2.2 | Secondary objectives of the trial |
• To compare efficacy of a single IV application of FCM with that of placebo on change of iron status on Day 56 (i.e., proportion of subjects with haemoglobin (Hb) ≥12 g/dL; serum-ferritin (s-ferritin) ≥50 ng/mL; transferrin saturation (TfS) >20%).
• To determine the relationship between change in iron status (s-ferritin and TfS) and improvement of fatigue symptoms.
• To compare the efficacy of a single IV administration of FCM with that of placebo in improving cognitive function (attention, concentration and short-term memory).
• To assess the safety of single IV administration of FCM. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Signed informed consent prior to study specific procedures.
2. Premenopausal, regularly menstruating women.
3. Age ≥18 years.
4. Body weight between 50 and 90 kg.
5. Haemoglobin ≥115 g/L.
6. Iron deficiency at screening defined as follows: − S-ferritin level <50 ng/mL, AND, TfS <20%, OR, − S-ferritin level <15 ng/mL.
7. Serum C-reactive protein: − <5 mg/L if not on oral contraception, OR, − <20 mg/L if use of oral contraception.
8. Minimum total score of 5 on the Piper Fatigue Scale (PFS) (mean of items 2 to 23).
9. Negative pregnancy test (serum human chorionic gonadotropin (hCG) at screening.
10. Normal levels of vitamin B12 and folic acid at screening.
11. Adequate contraception during the study period and for 1 month following study completion.
12. Availability and willingness to complete all study visits and procedures per protocol. |
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E.4 | Principal exclusion criteria |
1. Haemoglobin level <115 g/L.
2. Haemoglobinopathy.
3. Haemochromatose.
4. Major depressive disorder based on Patient Health Questionnaire (PHQ-9) (5 items with scores ≥2; one of which corresponds to question number 1 or 2).
5. Any active or unstable concurrent medical condition (e.g., cancer, renal dysfunction, liver dysfunction (aspartate aminotransferase (AST); alanine aminotransferase (ALT) >3-fold upper limit), angina (Class IV).
6. Known human immunodeficiency virus/acquired immunodeficiency syndrome, hepatitis B virus or hepatitis C virus infection.
7. Chronic inflammatory disease (e.g., rheumatoid arthritis; inflammatory bowel disease).
8. Documented history of clinically significant level of sleep apnoea defined as 5 or more episodes per hour of any type of apnoea.
9. Intake of concurrent medications that could interfere with physical or mental performance (e.g., antidepressive, antihistamines, narcotic or any chemotherapeutic agents known to cause drowsiness).
10. Important recent weight loss (>10% within the past month).
11. Body weight <50 kg or >90 kg.
12. Thyroid dysfunction, thyroid stimulating hormone >4 μU/mL.
13. Intake of iron preparations 4 weeks prior to screening.
14. Use of gestagens e.g., Implanon® Mirena®, Depo- Provera® for menstruation repression (see Section 7.7, Prohibited Therapy or Concomitant Treatment, page 35).
15. Known hypersensitivity to FCM or to any other iron preparation.
16. Pregnancy (positive hCG test at screening) or breast feeding.
17. Participation in any other interventional trial within 4 weeks prior to screening.
18. Inability to fully comprehend and/or perform study procedures or provide written consent in the Investigator’s opinion.
19. Subject is not using adequate contraceptive precautions during the study and for up to 1 month after the last dose of the study medication. A highly effective method of birth control is defined as those which result in a low failure rate (i.e., less than 1% per year) when used consistently and correctly such as implants, injectables, combined oral contraceptives, some intra-uterine devices, sexual abstinence or vasectomised partner.
20. Subject previously has entered this study.
21. Subject will not be available for follow-up assessments. |
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E.5 End points |
E.5.1 | Primary end point(s) |
The proportion of responders defined as subjects who have a decrease in total score of PFS (mean of items 2 to 23) of at least 1 point from baseline on Study Day 56. |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | No |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | Yes |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | Yes |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | Yes |
E.8.2.3 | Other | No |
E.8.3 |
The trial involves single site in the Member State concerned
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.4.1 | Number of sites anticipated in Member State concerned | 8 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 17 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
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E.8.7 | Trial has a data monitoring committee | No |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
|
A follow-up call will be made 2 weeks after the last study visit to record the status of any ongoing AEs from Visit 5. |
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 1 |
E.8.9.1 | In the Member State concerned months | 3 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 1 |
E.8.9.2 | In all countries concerned by the trial months | 3 |
E.8.9.2 | In all countries concerned by the trial days | 0 |